AL AmyloidosisNCT07585331
Towards Effective, Patient-tailored Anti-plasma Cell Therapies in AL Amyloidosis: Predicting Drug Response and Overcoming Drug Resistance
An observational study of AL Amyloidosis, run by Fondazione IRCCS Policlinico San Matteo di Pavia at 1 site in Italy.
Trial summary
Through an observational clinical study, partly prospective and partly retrospective, based on the collection of clinical data and on in vitro experimental analyses carried out on residual biological samples obtained during routine clinical procedures, it is proposed to identify predictive biomarkers of in vivo… Read more about this study
- Who is it for?
- Ages 18 to 99 years
- Conditions the study is for
- AL Amyloidosis
- How many people
- 250 participants
- Where
- 1 siteItaly
- Study dates
- 13 Jan 2022to 30 Mar 2026
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 99 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Newly diagnosed, treatment naïve AL amyloidosis patients with non-IgM plasma cell clones, evaluated at the amyloidosis Research and Treatment Center of Pavia, undergoing a diagnostic bone marrow aspiration will be enrolled.
Who may be able to join
- Biopsy-proven systemic AL amyloidosis
- No IgM clone
- No history of anti-plasma cell therapy
- Diagnostic bone marrow aspiration at ARTC
- Age > 18 years
- Willingness to allow use of clinical data and diagnostic leftovers of clinical specimens for research purposes through signing a written informed consent.
Who cannot join
- Non-AL amyloidosis
- IgM clone
- Previous anti-plasma cell therapy
- Age <18 years
- Failure to show willingness to allow use of clinical data and diagnostic leftovers of clinical specimens for research purposes.
Trial locations and contacts
1 site in 1 country.
About this study
Through an observational clinical study, partly prospective and partly retrospective, based on the collection of clinical data and on in vitro experimental analyses carried out on residual biological samples obtained during routine clinical procedures, it is proposed to identify predictive biomarkers of in vivo response to first-line therapies.
- Conditions
- AL Amyloidosis
- Medical subject headings
- Immunoglobulin Light-chain Amyloidosis
What is being measured
The primary outcome is the main question the study is designed to answer.
Identification of predictive biomarkers of response to proteasome inhibitor-based first-line therapy in AL amyloidosis
To identify and validate biological, genetic, and proteomic biomarkers predictive of in vivo response to proteasome inhibitor-based first-line therapies in patients with AL amyloidosis. This will be achieved through the integration of ex vivo drug sensitivity assays performed on patient-derived CD138+ plasma cells, characterization of plasma cell and mesenchymal stromal cell biological features, and retrospective and prospective molecular analyses. Biomarker profiles will be correlated with hematologic response at 6 months and used to develop a predictive statistical model of treatment response.
Measured: From baseline (diagnosis) to 6 months after initiation of first-line therapy
Secondary outcomes (3)
Molecular characterization of mechanisms of resistance to proteasome inhibitors
To investigate the mutational, transcriptional, and proteomic profiles associated with in vivo resistance to proteasome inhibitors by comparing plasma cell clones at diagnosis and minimal residual disease (MRD). Analyses will include next-generation sequencing, targeted DNA sequencing of genes involved in proteostasis, and quantitative proteomics to identify molecular determinants of drug resistance.
Measured: From baseline (diagnosis) to MRD assessment after achievement of complete hematologic response (approximately up to 12-24 months)
Evaluation of the therapeutic potential of deubiquitinating enzyme (DUB) inhibitors
To assess the ex vivo sensitivity of patient-derived amyloidogenic plasma cells to DUB inhibitors and to characterize the expression of DUB family members at RNA and protein level. The study will evaluate whether DUB inhibition can overcome resistance to proteasome inhibitors and identify candidate DUB inhibitors for therapeutic development.
Measured: At baseline (sample collection at diagnosis) and during ex vivo experimental analyses
Role of mesenchymal stromal cells in modulating drug response
To evaluate the contribution of patient-derived mesenchymal stromal cells to resistance against proteasome inhibitors by assessing their protective effect in co-culture systems with amyloidogenic plasma cells or cell lines.
Measured: At baseline (sample collection at diagnosis) and during ex vivo experimental analyses
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 May 2026
- Study start13 Jan 2022
- Primary completion30 Mar 2026
- Study completion30 Mar 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for AL AmyloidosisStudies of the same condition, open and under way.
- More trials run by Fondazione IRCCS Policlinico San Matteo di PaviaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 250 participants (actual)
Sponsor and investigators
- Lead sponsor
- Fondazione IRCCS Policlinico San Matteo di Pavia
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Mario Nuvolone, Fondazione IRCCS Policlinico San Matteo di Pavia
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07585331
- Sponsor's protocol ID
- P_52345_2019
- Status last verified
- May 2026
- First submitted
- 7 May 2026
- Last updated
- 13 May 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Towards Effective, Patient-tailored Anti-plasma Cell Therapies in AL Amyloidosis: Predicting Drug Response and Overcoming Drug Resistance
- ClinicalTrials.gov ID
- NCT07585331
- Acronym / sponsor ID
- P_52345_2019
- Conditions
- AL Amyloidosis
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 250 participants (actual)
- Lead sponsor
- Fondazione IRCCS Policlinico San Matteo di PaviaUniversity, hospital or other
- Study start
- 13 Jan 2022 (actual)
- Primary completion
- 30 Mar 2026 (actual)
- Study completion
- 30 Mar 2026 (actual)
- First posted
- 13 May 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Through an observational clinical study, partly prospective and partly retrospective, based on the collection of clinical data and on in vitro experimental analyses carried out on residual biological samples obtained during routine clinical procedures, it is proposed to identify predictive biomarkers of in vivo response to first-line therapies.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 18 to 99 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Newly diagnosed, treatment naïve AL amyloidosis patients with non-IgM plasma cell clones, evaluated at the amyloidosis Research and Treatment Center of Pavia, undergoing a diagnostic bone marrow aspiration will be enrolled.
Key inclusion criteria (6 listed)
- Biopsy-proven systemic AL amyloidosis
- No IgM clone
- No history of anti-plasma cell therapy
- Diagnostic bone marrow aspiration at ARTC
- Age > 18 years
Key exclusion criteria (5 listed)
- Non-AL amyloidosis
- IgM clone
- Previous anti-plasma cell therapy
- Age <18 years
- Failure to show willingness to allow use of clinical data and diagnostic leftovers of clinical specimens for research purposes.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Identification of predictive biomarkers of response to proteasome inhibitor-based first-line therapy in AL amyloidosis
Time frame: From baseline (diagnosis) to 6 months after initiation of first-line therapy
To identify and validate biological, genetic, and proteomic biomarkers predictive of in vivo response to proteasome inhibitor-based first-line therapies in patients with AL amyloidosis. This will be achieved through the integration of ex vivo drug sensitivity assays performed on patient-derived CD138+ plasma cells, characterization of plasma cell and mesenchymal stromal cell biological features, and retrospective and prospective molecular analyses. Biomarker profiles will be correlated with hematologic response at 6 months and used to develop a predictive statistical model of treatment response.
3 secondary outcomes
Molecular characterization of mechanisms of resistance to proteasome inhibitors
Time frame: From baseline (diagnosis) to MRD assessment after achievement of complete hematologic response (approximately up to 12-24 months)
Evaluation of the therapeutic potential of deubiquitinating enzyme (DUB) inhibitors
Time frame: At baseline (sample collection at diagnosis) and during ex vivo experimental analyses
Role of mesenchymal stromal cells in modulating drug response
Time frame: At baseline (sample collection at diagnosis) and during ex vivo experimental analyses
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 May 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of AL Amyloidosis, run by Fondazione IRCCS Policlinico San Matteo di Pavia at 1 site in Italy.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 13 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Italy. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Identification of predictive biomarkers of response to proteasome inhibitor-based first-line therapy in AL amyloidosis (time frame: From baseline (diagnosis) to 6 months after initiation of first-line therapy). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 May 2026.